Not applicableNo Longer AvailableNCT02233257What this trial is testingExpanded Access for Lorenzo's Oil (GTO/GTE) in AdrenoleukodystrophyWho this might be right forX-linked Adrenoleukodystrophy University of Minnesota
Not applicableEnded earlyNCT04008849What this trial is testingA Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456Who this might be right forInherited Metabolic Disorders (IMD) Magenta Therapeutics, Inc. 3
Not applicableLooking for participantsNCT04880356What this trial is testingLongitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.Who this might be right forInherited DiseaseRare DiseasesMetabolic Disease+3 more Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 100
Early research (Phase 1)WithdrawnNCT02410239What this trial is testingMT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)Who this might be right forCerebral Adrenoleukodystrophy Masonic Cancer Center, University of Minnesota
Large-scale testing (Phase 3)Study completedNCT00007020What this trial is testingCompassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic AcidWho this might be right forInfantile Refsum's DiseaseZellweger SyndromeAdrenoleukodystrophy+2 more Mirum Pharmaceuticals, Inc. 85
Not applicableLooking for participantsNCT05939232What this trial is testingRegistry of X-linked AdrenoleukodystrophyWho this might be right forX-linked Adrenoleukodystrophy Beijing Tiantan Hospital 200
Large-scale testing (Phase 3)UnknownNCT03143049What this trial is testingPomalidomide-Cyclophosphamide-Dexamethasone (PCD) Versus Pomalidomide-Dexamethasone (PD) in Relapse or Refractory MyelomaWho this might be right forRelapse Multiple Myeloma National University Hospital, Singapore 120
Large-scale testing (Phase 3)Study completedNCT03852498What this trial is testingA Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)Who this might be right forCerebral Adrenoleukodystrophy (CALD) Genetix Biotherapeutics Inc. 35
Large-scale testing (Phase 3)Study completedNCT01838941What this trial is testingBetaine and Peroxisome Biogenesis DisordersWho this might be right forPeroxisome Biogenesis Disorders McGill University Health Centre/Research Institute of the McGill University Health Centre 12
Large-scale testing (Phase 3)Study completedNCT02961803What this trial is testingMD1003-AMN MD1003 in AdrenomyeloneuropathyWho this might be right forAdrenomyeloneuropathyAdrenoleukodystrophyAMN MedDay Pharmaceuticals SA 67
Not applicableLooking for participantsNCT06796920What this trial is testingEpidural Spinal Cord Stimulation for Lower-limb Impairment in AdrenomyeloneuropathyWho this might be right forAdrenomyeloneuropathy Without Cerebral Involvement Third Military Medical University 10
Large-scale testing (Phase 3)Study completedNCT00176904What this trial is testingStem Cell Transplant for Inborn Errors of MetabolismWho this might be right forAdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more Masonic Cancer Center, University of Minnesota 135
Testing effectiveness (Phase 2)WithdrawnNCT03196765What this trial is testingSafety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With AdrenoleukodystrophyWho this might be right forX-Linked Adrenoleukodystrophy NeuroVia, Inc.
Early research (Phase 1)Temporarily pausedNCT06030648What this trial is testingMT2022-01: MSCs for ALDWho this might be right forCerebral Adrenoleukodystrophy Masonic Cancer Center, University of Minnesota 10
Large-scale testing (Phase 3)Looking for participantsNCT06513533What this trial is testingDimethyl Fumarate in AdrenomyeloneuropathyWho this might be right forAdrenomyeloneuropathy Without Cerebral Involvement Pujol, Aurora, M.D. 40
Testing effectiveness (Phase 2)Study completedNCT03406962What this trial is testingMGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)Who this might be right forInherited Metabolic Disorders (IMD) Magenta Therapeutics, Inc. 8
Not applicableUnknownNCT00278044What this trial is testingClinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese ChildrenWho this might be right forAdrenoleukodystrophy Far Eastern Memorial Hospital 20
Not applicableStudy completedNCT00538109What this trial is testingAn Open-Label, Multicenter, Expanded Access Study of Oral AMN 107 in Adult Patients With Imatinib (Glivec®/Gleevec®_ - Resistant or Intolerant Chronic Myeloid Leukemia in Blast Crisis, Accelerated Phase or Chronic PhaseWho this might be right forLeukemia, Myeloid, Chronic King Faisal Specialist Hospital & Research Center 8
Testing effectiveness (Phase 2)Study completedNCT00762632What this trial is testingCombination of Nilotinib (AMN107) and RAD001 in Patients With Acute Myeloid LeukemiaWho this might be right forAcute Myeloid Leukemia Technical University of Munich 40
Not applicableLooking for participantsNCT06224413What this trial is testingParticipants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene AutotemcelWho this might be right forCerebral Adrenoleukodystrophy (CALD) Genetix Biotherapeutics Inc. 120